Glepaglutide

Ironwood Pharmaceuticals Faces Setback as FDA Requires Additional Phase 3 Trial for Apraglutide

a month ago

• Ironwood Pharmaceuticals' stock plummeted by 37% after the FDA required a confirmatory Phase 3 trial for apraglutide, a treatment for short bowel syndrome with intestinal failure. • Pharmacokinetic analysis revealed that exposure and dosing in the original STARS Phase 3 trial were lower than intended due to preparation and administration issues. • Despite promising efficacy data showing 27 patients achieving enteral autonomy in the long-term extension trial, the company has engaged Goldman Sachs to explore strategic alternatives.

Ironwood Pharmaceuticals Faces Setback in Apraglutide Development for Short Bowel Syndrome

a month ago

• Ironwood Pharmaceuticals has announced that a confirmatory Phase 3 trial will be required for apraglutide in short bowel syndrome with intestinal failure, following its $1 billion acquisition of VectivBio. • The GLP-2 analogue apraglutide was positioned as a potential blockbuster drug with projected annual sales of $1 billion, offering advantages over existing treatments like Takeda's Gattex through improved potency and weekly dosing. • Ironwood has engaged Goldman Sachs to explore strategic alternatives for the company while continuing the long-term extension study and working with the FDA on the design of the confirmatory trial.

GI Advances: FDA Approvals, Novel Research Shape Gastroenterology in Early 2025

5 months ago

• The FDA approved mirikizumab (Omvoh) for Crohn's disease, marking its second approval in IBD after ulcerative colitis, based on positive Phase 3 trial results. • A tentative FDA approval was granted for a generic version of rifaximin for IBS-D, offering a potential cost-effective alternative pending the resolution of ongoing litigation. • Ironwood initiated a rolling NDA submission for apraglutide, aiming to address short bowel syndrome by reducing dependence on parenteral support. • Research indicates fecal microbiota transplantation (FMT) shows promise in treating type 1 diabetes and gastroenteropathy, improving patient quality of life.

Ionis' Tryngolza Approved for FCS as Arrowhead's Plozasiran Awaits FDA Review

5 months ago

• The FDA has approved Ionis Pharmaceuticals' Tryngolza (olezarsen) as the first treatment for adults with familial chylomicronemia syndrome (FCS). • Tryngolza, an RNA-targeted therapy, significantly reduces triglyceride levels and the risk of acute pancreatitis in FCS patients when used with a low-fat diet. • Arrowhead Pharmaceuticals' plozasiran, another RNA interference therapeutic for FCS, has been accepted for FDA review, with a decision expected by November 2025. • Clinical trials showed Tryngolza achieved a 57% placebo-adjusted mean reduction in triglycerides at 12 months, while plozasiran demonstrated an 80% median reduction.

FDA Issues Complete Response Letter for Zealand Pharma's Glepaglutide in Short Bowel Syndrome

5 months ago

• The FDA issued a Complete Response Letter for Zealand Pharma's glepaglutide, a GLP-2 analog, intended for short bowel syndrome (SBS) treatment, citing insufficient evidence of efficacy and safety. • The FDA recommends Zealand Pharma conduct an additional clinical trial to provide further evidence to confirm the efficacy and safety of glepaglutide at the to-be-marketed dose. • Zealand Pharma plans to initiate a Phase 3 trial in 2025 to support marketing authorizations outside the U.S. and EU and provide confirmatory evidence for U.S. resubmission. • Glepaglutide had orphan drug designation and showed promise in Phase 3 trials for reducing parenteral support in SBS patients, particularly with twice-weekly dosing.

FDA Gears Up for Year-End Decisions on Novel Therapies for Rare Diseases and Cancer

5 months ago

• The FDA is set to decide on Ionis Pharmaceuticals' olezarsen for familial chylomicronemia syndrome (FCS), a rare genetic disease, by December 19, following positive Phase III results. • Lexicon Pharmaceuticals awaits the FDA's decision on sotagliflozin as an adjunct therapy for type 1 diabetes by December 20, despite concerns raised by an advisory committee. • AstraZeneca and Daiichi Sankyo's Dato-DXd for non-squamous NSCLC is under FDA review, with a decision expected by December 20, based on Phase III data showing progression-free survival benefits. • Neurocrine Biosciences anticipates FDA decisions on crinecerfont for congenital adrenal hyperplasia (CAH) by December 29 and 30, potentially marking the first new treatment in 70 years.

FDA Wraps Up 2024 with Key Approvals for Drugs Targeting Various Conditions

6 months ago

• The FDA approved Vertex's Alyftrek for cystic fibrosis, offering improved dosing and potential market exclusivity. • Novo Nordisk's Alhemo was approved for hemophilia A and B, providing a new option for patients with inhibitors. • Bristol Myers Squibb's Opdivo Qvantig gained approval as a subcutaneous formulation, offering faster administration for various solid tumors. • Eli Lilly's Zepbound secured approval for obstructive sleep apnea in obese adults, marking the first prescription medicine for this condition.

Zealand Pharma and Palatin Technologies Advance Obesity and Metabolic Disease Pipelines

6 months ago

• Zealand Pharma reported positive Phase 1b results for petrelintide and dapiglutide, advancing both into Phase 2b trials for overweight and obese patients. • Boehringer Ingelheim's survodutide, a Zealand Pharma-partnered asset, showed positive Phase 2 results in MASH and progresses to Phase 3 trials. • Palatin Technologies anticipates topline results from its Phase 2 BMT-801 trial of bremelanotide with tirzepatide for obesity in Q1 2025.

Cell Therapy and Targeted Therapies Dominate Oncology Advances in Early 2025

7 months ago

• The FDA issued a CRL for Atara Biotherapeutics' tabelecleucel due to third-party manufacturing issues, not efficacy or safety data, delaying potential approval for EBV+ PTLD. • EsoBiotec dosed the first patient in a trial for ESO-T01, an in vivo BCMA-directed CAR-T therapy for multiple myeloma, aiming for lower costs and simplified administration. • Obecabtagene autoleucel (obe-cel) gained FDA approval for relapsed/refractory B-cell precursor ALL, offering a less toxic CD19-directed CAR T-cell therapy option. • Arlocabtagene autoleucel (arlo-cel) shows promise in heavily pretreated relapsed/refractory multiple myeloma, eliciting a 48% complete response rate in phase 1 studies.

Drug Development Updates